Gene Therapy Going Strong

Gene Therapy Collaborative Group Targets Neuromuscular Disease

At Nationwide Children’s Hospital, gene therapy strategies to reverse or prevent damage caused by muscle-wasting diseases are gaining strength, especially in animal models of human disease. Through The Ohio State University and Nationwide Children’s Muscle Group, 24 laboratories at Nationwide Children’s and The Ohio State University are working to improve approaches to treating muscle injury and disease.

“Research in muscle biology is intensely comprehensive incorporating aspects of development, physiological function, metabolism, exercise, aging, and disease,” said Denis Guttridge, PhD, of OSU’s Department of Molecular Virology, Immunology and Medical Genetics and one of the Muscle Group coordinators. “It has become increasing important in one’s research to try to understand as many of these concepts as possible, but doing so as an individual laboratory can be quite demanding.”  Dr. Guttridge says the advantage of the Muscle Group is that each of the 24 participating laboratories has its own unique expertise in muscle biology. “Working together and sharing our respective expertise has naturally improved the quality of the science that each of members have performed,” he said.

The most notable advances are being made in the group’s research into muscular dystrophy and spinal muscular atrophy (SMA), two of the most common and devastating neuromuscular diseases.  “The most promising things we’ve found are that in pre-clinical models we can correct the gene defect in these diseases, deliver genes through the circulatory system, help build larger and stronger muscle, and deal with the immune responses caused by gene delivery,” said Jerry Mendell, MD, director of the Center for Gene Therapy at The Research Institute and a Muscle Group coordinator.  “There is still much work to be done to transform our techniques into applicable interventions in children, but gene therapy as a successful treatment for Duchenne muscular dystrophy and spinal muscular atrophy is on the horizon.”

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